EURORDIS-Rare Diseases Europe

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EURORDIS-Rare Diseases Europe

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An alliance of patient organisations improving the lives of all people living with rare diseases.

04/11/2026

🇪🇺 Europe has led the way on rare diseases before. With an EU Action Plan, it can lead again.

Yesterday, at The Parliament Magazine's Health Summit in Brussels, EURORDIS Board member Simona Bellagambi presented our recently launched European Blueprint for Rare Diseases, setting out how Europe can turn its ambitions into meaningful improvements for patients.

During her intervention, Simona highlighted three priorities:

🔹 Europe has built strong foundations, including the European Reference Networks, Orphanet and ERDERA. But inequalities in diagnosis, specialised care and access to treatments persist.

🔹 Innovation and health equity must go hand in hand. EU initiatives such as the Biotech Act can advance research and development, but an overarching EU Action Plan is needed to connect innovation with patients' needs.

🔹 The Blueprint provides a roadmap for action, with 18 priorities across six areas, developed by patients and other stakeholders. With growing political momentum, the European Commission and Member States must now act.

And the conversation continues today at the European Parliament!

In just 30 minutes, our colleague Inés Hernando will contribute to the Intergroup on Cancer and Rare Diseases Expert Dialogue on Cross-border Healthcare, discussing how EU rules can improve patients' access to specialised care across borders, another priority reflected in our Blueprint.

👉 Watch the discussion live: https://go.eurordis.org/Iu3MSe

📢 Stay tuned! We'll be sharing the main takeaways from the event, including Inés' contribution to the discussion.

European Cancer Organisation SIOP Europe, the European Society for Paediatric Oncology - SIOPE

03/11/2026

💊 Over the past two days in Paris, patient advocates came together for the REMEDi4ALL Repurposing Bootcamp for Patient Representatives!

The focus? Exploring how existing medicines could offer new treatment opportunities and, importantly, how patients can help make that happen.

Through this hands-on training, combining expert-led sessions, practical workshops and real-world case studies, participants gained the knowledge and tools to:

🔎 Identify promising opportunities for drug repurposing.
🧬 Develop strategies to take an idea from initial concept towards patient access.
🤝 Evaluate and fund projects with the potential to make a real difference

But beyond the technical knowledge, the Bootcamp was also an opportunity to exchange experiences, build connections and strengthen patient advocates' role in drug repurposing research.

As a partner in REMEDi4ALL, we're glad to have contributed to an initiative that helps patient representatives turn new knowledge into practical action and play a more active role in shaping treatment opportunities.

👉 Learn more about the REMEDi4ALL initiative: https://go.eurordis.org/Remedi4all

03/11/2026

🧬 What would make clinical trials more accessible for people living with rare diseases?

Sometimes, the biggest challenges aren't just about the treatment being studied. They're about whether taking part in a trial is possible in the first place.

Your experience can help change that.

Together with RealiseD, EURORDIS has co-created a survey to better understand what makes clinical trial participation difficult for people living with rare and ultra-rare diseases, and what could make research more inclusive.

💬 Living with a rare disease, or supporting someone who is? We want to hear from you, whether or not you've participated in a clinical trial before.

🌍 Available in over 15 languages and open to patients, caregivers and family members. Share your perspective: https://go.eurordis.org/RealiseDSurvey

02/11/2026

✨ Some achievements deserve more than a round of applause. They deserve a night to remember. And on 15 February 2027, we're bringing the rare disease community together in Brussels to do just that!

Registrations are officially OPEN for the 16th EURORDIS Black Pearl Awards! 🎉

Every year, the Black Pearl Awards shine a spotlight on the people and organisations who are changing what it means to live with a rare disease. The patient advocates who refuse to give up. The researchers pushing boundaries. The changemakers turning ideas into real progress.

But this evening is about more than recognising achievements. It's a chance to come together, celebrate how far we've come, share experiences and enjoy a special night of inspiring stories and artistic performances.

👉 Secure your place now: https://go.eurordis.org/BPA2027registrations

📅 Monday, 15 February 2027
📍 Brussels, Belgium

28/10/2026

The World Orphan Drug Congress Europe 2026 has wrapped up in Amsterdam!

Over the past three days, patients, policymakers, researchers and industry representatives came together to discuss the future of rare disease research, drug development and access to treatments.

Our EURORDIS colleagues brought the patient perspective to several key discussions.

💊 Our CEO Jean Saslawsky joined two keynote panels, emphasising that patients must be meaningfully involved in shaping EU policies and medicines development. He highlighted good practices including the EURORDIS Charter for Clinical Trials in Rare Diseases, Community Advisory Boards and the ACT-EU initiative, alongside the need for improved patient retention in clinical trials, reporting, patient-focused registries and funding for patient participation in research. He also stressed the potential of rare diseases to drive innovation and European competitiveness, calling for greater EU policy coordination.

🔎 Gulcin Gumus, our Senior Research and Policy Manager, contributed to discussions on earlier diagnosis, highlighting the importance of patient partnership and co-designed diagnostic pathways in shaping research, healthcare and policy. She also moderated a panel exploring the health and economic benefits of earlier diagnosis.

🇪🇺 At the closing panel, Valentina Bottarelli, our Head of Policy and Public Affairs, presented key proposals from the European Blueprint for Rare Diseases, highlighting the rare disease community's priorities for a future EU Action Plan.

Together, these discussions reinforced the need for a coordinated approach that places patients at the heart of decisions affecting their lives.

This is central to our call for a that delivers meaningful improvements for the 30 million people living with rare diseases across Europe.

👏 Thank you to everyone who contributed!

👉 Explore the European Blueprint for Rare Diseases: https://go.eurordis.org/q0y6jc

27/10/2026

📸 What does living with a rare disease look like through your lens?

Submissions are officially open for the EURORDIS Photo Award 2027!

Behind every photograph is a story worth sharing. A moment of everyday life, a challenge, a connection, a celebration - whatever captures the reality of living with a rare disease.

🌍 Open to participants worldwide, across all rare diseases, the Photo Award is an opportunity to share these experiences with the rare disease community and beyond.

A selection of finalists will be chosen by award-winning photographer Marcus Bleasdale, before the public gets to vote for their favourites.

🏆 The winners will be announced at the 16th EURORDIS Black Pearl Awards on 15 February 2027.

Your story deserves to be seen. Will you share it with us?

👉 Explore last year's winning photos and submit yours today: https://go.eurordis.org/submityourphoto

26/10/2026

🗣️ Be part of the conversation on the future of rare disease research!

On 13 November, “Research as Care: Why Rare Diseases Matter for Europe's Future” will bring together policymakers, researchers, healthcare professionals, industry representatives and patient organisations to explore how research and innovation can become a standard part of rare disease care.

Co-organised by Rare Diseases Ireland and EURORDIS, this Associated Event of Ireland's Presidency of the Council of the European Union will connect perspectives from across Europe, with patient partnerships at the heart of the discussions.

📍 Radisson Blu Royal Hotel, Dublin
📅 Friday, 13 November 2026
💻 In-person and online participation available

👉 Registration is still open, don't miss out: https://go.eurordis.org/rare-diseases-event

Debra Ireland - The Butterfly Skin Charity | Neurological Alliance of Ireland (NAI) | NCBRS Worldwide Foundation | Irish Platform for Patient Organisations, Science, Industry

24/10/2026

A vision is not enough. Europe needs a plan to make it happen. That's exactly what the European Blueprint for Rare Diseases sets out to do.

But what is the Blueprint, and what does it actually propose?

Think of it as a roadmap for a future EU Action Plan on Rare Diseases. Not just a list of ambitions, but a proposal for how Europe can turn its existing efforts into coordinated, measurable action.

Its essential building blocks include:

🎯 Shared goals and clear targets to drive progress.
🤝 Joint governance involving EU institutions, Member States and the rare disease community.
💶 Dedicated resources and aligned funding to support delivery.
📊 Monitoring and accountability to measure progress.

And crucially, patients as partners throughout the process.

Co-created over ten months by patients, clinicians, researchers, public authorities and industry across Europe, our call to the European Commission is clear: bring forward an EU Action Plan on Rare Diseases that builds on these proposals.

The rare disease community has set out the way forward. Now, we need the political commitment to turn it into action.

👉 Discover the European Blueprint for Rare Diseases: https://go.eurordis.org/PressRelease-Blueprint

22/10/2026

⏳ You have only 11 hours left to register!

On 4 November, Giorgos Georgiou will be joined by fellow MEPs Tilly Metz, Tomislav Sokol and Marta Temido for the Intergroup's Expert Dialogue on Cross-Border Healthcare at the European Parliament.

From access to specialised expertise and clinical trials to strengthening patients' rights, the Expert Dialogue on Cross-Border Healthcare will explore what needs to change as Europe considers modernising its rules.

📅 4 November 2026📍 European Parliament, Brussels

🚨 Registration closes at midnight on 22 October! Places are limited, and advance registration is mandatory for all in-person attendees, including badge holders.

👉 Join us in Brussels – register before the deadline: https://go.eurordis.org/cross-border-expert

European Cancer Organisation | SIOP Europe, the European Society for Paediatric Oncology - SIOPE

22/10/2026

🔬 45 years. That's the median time between the first scientific publication and the first orphan drug designation for rare diseases that reach this milestone.

A striking finding from a new study published in the Orphanet Journal of Rare Diseases, which exposes just how uneven progress remains across the rare disease landscape.

Developed through IRDiRC's PLUTO mission, the research reveals that half of the rare diseases analysed have scientific publications but no evidence of clinical development, while fewer than 10% have reached an orphan drug designation or marketing authorisation.

🤝 Our Head of Therapies & Access, Virginie Hivert, is among the co-authors of this research, helping shed light on the gaps that continue to shape rare disease research and treatment development.

Understanding where progress stalls is an important step towards ensuring that more rare diseases benefit from research and innovation. 👉 Read the full study: https://go.eurordis.org/NewArticle

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